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Cell profiling and efficacy evaluation in disease-relevant or patient-derived cell systems using the latest technologies.

Antisense oligonucleotide (Aso) screening SERVICES

Integrated Drug Discovery Services with Patient-Specific Disease Models for N-of-1 Solution

At iXCells, we believe everyone deserves a cure no matter how rare the disease is.

Our researchers and scientists combined in-depth understanding of cell engineering and RNA therapeutics are dedicated to help you accelerate your drug development, including individualized medicines. Our customized preclinical CRO services include patient iPSC reprogramming, cell line engineering, iPSC-neuron differentiation, and ASO / siRNA screening.

We help patients in the race against time. Our relationship with rare patients advocacies allow us to understand the uniqueness of each ultra-rare case and streamline our business logistics. With a patient-centric research support, we also help our customers and partners to exchange their experiences and resources during their journey in seeking the cure for rare diseases.

Together we can turn the impossible into miracles.

Some of our clients & partners

The right expertise to support your preclinical drug development

Cell Model Development

As the cell model expert, we are specialized in a variety of disease-relevant cell systems, including immortalized cell lines and iPSC-derived models. We help generate patient-specific iPSC lines and iPSC-neurons which are valuable tools to shorten your time in developing treatments for epilepsy and other neuronal disorders.

RNA Therapeutics

We have intensive experience in assay development, including gymnosis and transfection optimization in different cell systems. Our experience encompasses various clinical-grade ASO technologies including 2’MOE-, 2’O-Me-, LNA- and cEt-modified gapmer and steric blocking ASOs.

Patient Centricity

We understand each rare case is unique. Our patient-centric services enable foundation leaders and research partners to establish their preclinical studies based on disease progression, mode-of-action information, POC results, financial sources, etc.

Information Exchange

We believe that the joined forces of researchers, physicians, patient advocacy and regulatory professionals are critical to tackle rare diseases. We create a platform for our clients and partners share their experiences / resources and establish best practices along their journey in fighting devastating diseases.

Case study individualized aso drug development

1. Two test ASOs showed dose-dependent knock-down of GOI
2. Minimal effect of Negative control ASO reagents on Target Gene across screening plates
  • Each dot represents one biological sample (average of technical triplicates)
  • All samples are normalized to the average of non-treatment samples
  • All samples are normalized to the average of non-treatment samples
  • PC = Positive Control ASO
  • NC = Negative Control ASO

Aso Screening using patient-derived cell model

Our comprehensive Antisense Oligonucleotides (ASOs) screening services employ human (patient-derived) cell models. The iXCells scientific team have extensive experience with ASO in vitro screening using various disease relevant cellular models, including human iPSC-derived neurons and primary cells.
Antisense Oligonucleotides (ASOs) are single-stranded deoxynucleotide analogs, usually 15–20 bp in length. Their sequence is complementary to the sense sequence of target mRNA. ASOs can be used in traditional drug discovery workflows for target identification and validation and are now being explored as therapeutics themselves. ASO-based therapy is an active area of drug development designed to treat a variety of gene specific diseases, especially in the setting of personalized medicine and orphan diseases.